Targeted Transposons for Gene Therapy

Award Information
Agency:
Department of Health and Human Services
Branch
n/a
Amount:
$174,011.00
Award Year:
2007
Program:
STTR
Phase:
Phase I
Contract:
1R41EB007451-01
Agency Tracking Number:
EB007451
Solicitation Year:
n/a
Solicitation Topic Code:
n/a
Solicitation Number:
n/a
Small Business Information
DISCOVERY GENOMICS, INC.
DISCOVERY GENOMICS, INC., 614 MC KINLEY PL NE, MINNEAPOLIS, MN, 55413
Hubzone Owned:
N
Minority Owned:
N
Woman Owned:
N
Duns:
142797880
Principal Investigator:
PERRY HACKETT
(612) 656-4485
PERRYH@DISCOVERYGENOMICS.NET
Business Contact:
RSCOTT MCIVOR
() -
perryH@discoverygenomics.net
Research Institution:
UNIVERSITY OF MINNESOTA

UNIVERSITY OF MINNESOTA
100 CHURCH ST SE
MINNEAPOLIS, MN, 55455 3000

Nonprofit college or university
Abstract
DESCRIPTION (provided by applicant): Discovery Genomics, Inc. is focused on development of the Sleeping Beauty (SB) transposon system as a non-viral means of integrating new gene sequences in cells and tissues for therapeutic purposes. Here we propose to d evelop a method for improved targeting of the SB system to specific cell types following systemic injection by coupling a targeting tether to the plasmid carrier of the transposon. This approach will allow 'universal' vectors that could carry any gene and be directed to any cell type without modification of the transposase or transposon for each cellular target. This project will dovetail with DGI's ongoing SBIR project to deliver Factor VIII and IX genes in appropriate animal model systems. Thus, this proj ect has high-impact potential because the techniques we will develop can be used for treatment of multiple diseases using a 'universal' non-viral vector system whose properties will be applicable to many therapeutic transgenes. The Specific Aims of the pro ject are to: 1. Construct a Liver- directed Targeting Tether (LTT) vector system to increase uptake of SB transposons into liver cells. This aim will be accomplished by construction of a LTT targeting tether comprised of a LexA DNA-binding domain fused to a ligand specific for hepatocytes and construction of a plasmid, pKLAT2, that contains an SB transposon plus multimeric LexA operator sites to which the targeting tether can bind. 2: Evaluate the efficiency in cultured liver cells (HuH7 and HepG2) of a LTT targeting tether to enhance uptake and transposition of a pKLAT2 transposon vector. HeLa cells will serve as a control for cells that lack appropriate liver-specific receptors. 3: Evaluate the efficiency of a LTT targeting tether to enhance uptake and tra nsposition of a pKLAT2 transposon vector into liver cells in mice.

* information listed above is at the time of submission.

Agency Micro-sites


SBA logo

Department of Agriculture logo

Department of Commerce logo

Department of Defense logo

Department of Education logo

Department of Energy logo

Department of Health and Human Services logo

Department of Homeland Security logo

Department of Transportation logo

Enviromental Protection Agency logo

National Aeronautics and Space Administration logo

National Science Foundation logo
US Flag An Official Website of the United States Government